Eli Lilly Partners with Rznomics in $1.3B Deal to Develop RNA Editing Therapy for Hereditary Hearing Loss
• Rznomics Inc. has secured a potential $1.35 billion global license option agreement with Eli Lilly to codevelop an RNA editing-based gene therapy targeting hereditary hearing loss.
• Under the partnership structure, Rznomics will lead initial R&D efforts to discover and develop the therapy, while Lilly will handle subsequent development and commercialization phases.
• The deal represents a significant expansion of Lilly's gene therapy pipeline for hearing disorders, though specific details regarding up-front payments and option terms remain undisclosed.
ProQR Therapeutics Bolsters Leadership Team with Strategic CFO and CMO Appointments as RNA Editing Platform Advances to Clinical Stage
• ProQR Therapeutics has appointed Dennis Hom as CFO and Dr. Cristina Lopez Lopez as CMO to support the advancement of its Axiomer™ RNA editing technology platform into clinical development.
• Dennis Hom brings over 25 years of financial leadership experience, having raised more than $4.5 billion in capital and executed transactions exceeding $57 billion in value throughout his career.
• Dr. Cristina Lopez Lopez contributes 20+ years of global R&D leadership with expertise in translational science, previously serving as Global Head of Neurodegeneration at Johnson & Johnson.
Nanoscope's Optogenetic Gene Therapy Shows Promise in Retinitis Pigmentosa Clinical Trials
• Nanoscope Therapeutics' MCO-010 gene therapy demonstrated clinically meaningful vision improvements in 16 out of 18 patients with severe retinitis pigmentosa in a Phase 2 trial, compared to only four of nine in the placebo group.
• The mutation-agnostic therapy uses a synthetic opsin (MCO protein) delivered via intravitreal injection to restore light-detecting function in damaged retinal cells, potentially benefiting patients regardless of their specific genetic mutation.
• Following treatment, patients showed significant improvements in vision-guided mobility and object recognition, with some experiencing "transformative" results while maintaining a favorable safety profile with no serious adverse events reported.
RNA Therapy Market Set to Reach $4.16 Billion by 2034 as Clinical Trials Accelerate
• The global RNA therapy clinical trials market is projected to grow from $2.85 billion in 2024 to $4.16 billion by 2034, with a CAGR of 3.85%, driven by advancements in mRNA, siRNA, and antisense oligonucleotide-based therapies.
• Over 80 companies are currently evaluating more than 100 RNA therapies across various development stages, with significant activity in rare diseases, oncology, and genetic disorders.
• Recent breakthroughs include FDA clearance for the first CRISPR/Cas13 RNA-editing therapy for neovascular age-related macular degeneration and promising results for RNA therapies targeting rare muscular dystrophies.
CRISPR Weekly Roundup: Advancements in Gene Editing and Clinical Trials
• Beam Therapeutics reported a patient death in their BEAM-101 sickle cell disease trial, likely due to the conditioning regimen.
• AstraZeneca scientists engineered PsCas9 for therapeutic genome editing in mouse liver, showing promise for hypercholesterolemia treatment.
• YolTech Therapeutics' novel LNP system delivers base editor mRNA to bone marrow cells, activating foetal haemoglobin production for blood disorder treatment.
RNA Editing Shows Promise in Treating Genetic Conditions Where DNA Editing Falls Short
• Wave Life Sciences pioneered clinical RNA editing, treating alpha-1 antitrypsin deficiency (AATD) by correcting mRNA mutations, offering a novel therapeutic approach.
• RNA editing, utilizing ADAR enzymes and guide RNAs, allows for temporary and reversible modifications, reducing risks associated with permanent DNA alterations.
• Companies like Korro Bio and Ascidian Therapeutics are expanding RNA editing applications to target diseases like Parkinson’s, ABCA4 retinopathy, and certain cancers.
• Challenges remain in RNA editing, including improving specificity, managing transient effects, and enhancing delivery methods for broader clinical applications.
Wave Life Sciences' RNA Editing Data Sparks Rally in RNA Therapy Stocks
• Wave Life Sciences reported positive proof-of-mechanism data from a single-dose trial of its RNA-editing therapy for lung and liver diseases, causing its stock to surge.
• The trial data marks the earliest human data for any RNA editing therapy, validating the technology and exceeding analysts' expectations for early efficacy.
• Following Wave Life's announcement, shares of other RNA-editing companies, including ProQR Therapeutics and Korro Bio, also experienced significant gains.
• Wave Life plans to release data from a multi-dose trial of its RNA-editing therapy in 2025, further fueling optimism in the RNA-editing therapeutic space.
Eli Lilly Invests $4.5 Billion in Advanced Drug Manufacturing Facility
• Eli Lilly is investing $4.5 billion to establish the Lilly Medicine Foundry in Indiana for drug production and clinical trial medicine manufacturing.
• The facility will integrate advanced manufacturing technologies to enhance the production of diverse molecular therapies, including biologics and nucleic acids.
• This initiative aims to optimize processes, increase trial medicine capacity, reduce costs, and minimize environmental impact across Lilly's network.
• The investment builds on Lilly's previous expansions in Lebanon, Indiana, and supports the company's growing pipeline of new medicines.
Eli Lilly Acquires Akouos for $610M to Expand Gene Therapy Portfolio in Hearing Loss Treatment
• Eli Lilly has agreed to acquire hearing loss gene therapy developer Akouos for up to $610 million, with an initial payment of $487 million and potential milestone-based payments of up to $123 million.
• Akouos's lead candidate AK-OTOF, targeting otoferlin gene mutations in sensorineural hearing loss, has received FDA approval for clinical testing and could potentially benefit 200,000 patients worldwide.
• The acquisition strengthens Lilly's gene therapy portfolio and expands its presence in genetic medicine, following its previous $1 billion acquisition of Prevail Therapeutics in 2020.
Free Genetic Testing Initiative Accelerates Drug Development for Inherited Retinal Diseases
• The Foundation Fighting Blindness's My Retina Tracker program offers free genetic testing and counseling for inherited retinal disease patients in the US, having enrolled over 6,000 participants since 2017.
• ProQR Therapeutics becomes the first industry partner of the program, gaining access to crucial genotype/phenotype data to advance development of RNA-based therapies for specific genetic mutations.
• The initiative aims to test 20,000 patients in coming years, helping overcome key challenges in rare disease drug development including patient identification and clinical trial optimization.
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